It was January 2017, mere days after Spinraza was first approved to treat spinal muscular atrophy. A group of SMA parents, appalled with the lingering silence from UK officials, decided to take matters into their own hands and make a strong push for access to treatment as a recognised group. The name “TreatSMA” came up. A friendly graphics designer created the two-colour DNA–Broken Heart–Hourglass logo. An internet domain was registered and soon a temporary website was up. The group was quickly recognised by the National Institute for Health and Care Excellence as an official stakeholder in the Spinraza appraisal, a lengthy process which had just commenced.
By then, elsewhere in Europe hundreds of children with the most severe form of SMA were treated with Spinraza for free under Biogen’s Expanded Access Programme. Seeing the amazing efficacy of the new drug, Dr Laurent Servais, neurology head at a Paris hospital, decided with his team to treat as many children from all over Europe as they could. Soon a Poland-based SMA organisation funded an additional doctor in the French team to help with Spinraza administration.
Meanwhile in the UK, the NHS continued to refuse approving the Spinraza EAP. The officials kept repeating that SMA was not worth treating with Spinraza – it would be too risky, too costly, or too complicated. Children’s lives did not seem to matter. The TreatSMA team spoke to hospitals and went to meet the NHS decision makers at the highest levels. In parallel, we linked up with Paris and facilitated voyages to France for UK families. All in all, 13 severely ill UK children received life-saving injections in France. TreatSMA became a familiar name at home and beyond and our Facebook support group became the place to go to discuss all things SMA.
After seven long months, during which several children sadly passed away while waiting for treatment, and after intense pressure from TreatSMA, other SMA organisations and clinicians, NHS finally authorised Spinraza EAP in the UK. Our new website now tracked the UK hospitals as they were opening up to this breakthrough treatment.
The TreatSMA team then embarked on another task: to make sure that the regulators recommend Spinraza as a treatment for everyone with SMA. We engaged with Biogen and NICE, travelled to meetings, spoke at the Parliament, wrote opinions and comments, presented evidence of treatment efficacy from all over the world. We even became official NICE consultees. Finally, NICE agreed to review Spinraza as a treatment for all.
However, the process was slow, and NICE experts clearly weren’t keen on recommending Spinraza. The SMA Community took to the media. We reached out to MPs. Finally we all took to the streets. We were exasperated. We were determined. We fought for life.
Then last year TreatSMA hit an invisible wall. As we grew, we were lucky to receive generous donations. But now the law required us to account for funds in a very specific way. In order to keep up with the work, spirit and energy without breaching the law, we had to get TreatSMA registered as a charitable organisation.
It was not a decision we took lightly. Our aim always was to help other families in accessing care and treatment; not to create another charity. But soon it became obvious that we wouldn’t progress further without having a formal structure in place.
So, on 25th March 2019 TreatSMA was registered by the Charity Commission as a “charitable incorporated organisation governed by the laws of England and Wales”. We were now legally recognised. Our funds, frozen by PayPal since late 2018, were unfrozen. Our email system, earlier frequently blocked by Google, was converted to a grant-funded non-profit system. Now we could come up with new ideas and new projects and seek funding for them. Six trustees now make sure that we all work towards our goal written into our Constitution:
To promote and protect the health and wellbeing of persons with spinal muscular atrophy, their families and carers, in particular by:
- advancing knowledge in all aspects of spinal muscular atrophy;
- improving access to appropriate healthcare, including diagnostics, treatments, and assistive technologies; and
- promoting social inclusion of people with spinal muscular atrophy, their families and carers
Naturally, we are still the same bunch of SMA families eager to help others, who work voluntarily from their homes in their spare time. We are not a corporation. We can still be contacted via email, facebook, or our personal mobiles. But we are also looking into the future as an entity able to push for our cause more strongly and in a more co-ordinated way.
This long journey would not have been possible withouth the engagement of many healthcare professionals, regulators, our organisational partners, businesses, donors, and foremostly without the engagement of so many SMA families who share our common goal. Thank you, we will continue our journey together.
In the near future, as soon as we have settled in our new structure, we will share our ideas on the next steps, and ask for more. Let us mention that we have already started looking beyond Spinraza, to new upcoming treatments, routine newborn screening, home care, and equipment-related matters. Much more is to come, so please keep your fingers crossed for TreatSMA!
